Outcomes of Nononcology Accelerated Approvals (Reply)

Liu ITT, Kesselheim AS, Cliff ERS - JAMA

  • Regulation and Clinical Evidence

Summary:

Surrogate measures used for accelerated approval are, by definition, not fully validated and may lack strong evidence linking them to clinical benefit. The authors highlight recent cases where reliance on such surrogates led to regulatory withdrawals or ongoing uncertainty, and emphasize that while accelerated approval can speed access to new treatments, it also requires careful follow-up and realistic communication about risks.

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Recent Work from PORTAL

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How Congress Can Fix Expert Advisory Committees

Daval CJR, Kesselheim AS - STAT

  • Regulation and Clinical Evidence
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International Regulatory Status and Reimbursement Recommendations of Haematological Malignancy Drugs Withdrawn from the FDA Accelerated Approval Pathway

Mulsant SS, Kesselheim AS, Naci H - Journal of Cancer Policy

  • Price, Value, and Access
  • Regulation and Clinical Evidence
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Comments on FDA Discussion Paper, “Considerations for the Regulation of Generative AI-Enabled Medical Devices”

Jaiswal N, Gorijavolu R, Thomas S, Hwang TJ, Celi LA, Kesselheim AS - Food and Drug Administration (FDA)

  • Regulation and Clinical Evidence